Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.

@article{Leone2000AspartoacylaseGT,
  title={Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.},
  author={Paola Leone and Christopher G. Janson and Larissa L Bilaniuk and Zhimin Wang and F L. Sorgi and Leaf Huang and Reuben K Matalon and Rajinder Kaul and Zhiyuan Zeng and Andrew Freese and Scott W.J. McPhee and Edward Mee and Matthew John During},
  journal={Annals of neurology},
  year={2000},
  volume={48 1},
  pages={27-38}
}
With the ultimate goal of developing safe and effective in vivo gene therapy for the treatment of Canavan disease and other neurological disorders, we developed a non-viral lipid-entrapped, polycation-condensed delivery system (LPD) for central nervous system gene transfer, in conjunction with adeno-associated virus (AAV)-based plasmids containing recombinant aspartoacylase (ASPA). The gene delivery system was tested in healthy rodents and primates, before proceeding to preliminary studies in 2… CONTINUE READING
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